CN114466867A - 用于抑制α-突触核蛋白表达的锌指蛋白转录因子 - Google Patents

用于抑制α-突触核蛋白表达的锌指蛋白转录因子 Download PDF

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CN114466867A
CN114466867A CN202080069917.0A CN202080069917A CN114466867A CN 114466867 A CN114466867 A CN 114466867A CN 202080069917 A CN202080069917 A CN 202080069917A CN 114466867 A CN114466867 A CN 114466867A
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Chinese (zh)
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A.哈塔米
B.蔡特勒
L.张
D.E.帕肖恩
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN202080069917.0A 2019-10-02 2020-10-02 用于抑制α-突触核蛋白表达的锌指蛋白转录因子 Pending CN114466867A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962909496P 2019-10-02 2019-10-02
US62/909,496 2019-10-02
US202062959153P 2020-01-09 2020-01-09
US62/959,153 2020-01-09
PCT/US2020/054148 WO2021067871A1 (en) 2019-10-02 2020-10-02 Zinc finger protein transcription factors for repressing alpha-synuclein expression

Publications (1)

Publication Number Publication Date
CN114466867A true CN114466867A (zh) 2022-05-10

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ID=73014653

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CN202080069917.0A Pending CN114466867A (zh) 2019-10-02 2020-10-02 用于抑制α-突触核蛋白表达的锌指蛋白转录因子

Country Status (10)

Country Link
US (1) US20220324928A1 (ko)
EP (1) EP4041754A1 (ko)
JP (1) JP2022551286A (ko)
KR (1) KR20220071248A (ko)
CN (1) CN114466867A (ko)
AU (1) AU2020356962A1 (ko)
CA (1) CA3156258A1 (ko)
IL (1) IL291827A (ko)
TW (1) TW202126681A (ko)
WO (1) WO2021067871A1 (ko)

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Zinc finger protein derivatives and methods therefor
ATE407205T1 (de) 1994-08-20 2008-09-15 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
EP1060261B1 (en) 1998-03-02 2010-05-05 Massachusetts Institute of Technology Poly zinc finger proteins with improved linkers
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
CA2542232A1 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals, Inc. Method for treating neurodegenerative disease by inhibiting alpha-synuclein
EP2357226B1 (en) 2005-10-18 2012-03-14 Precision Biosciences Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
KR101974036B1 (ko) 2010-05-03 2019-04-30 상가모 테라퓨틱스, 인코포레이티드 아연 핑거 모듈을 연결하기 위한 조성물
WO2012094132A1 (en) * 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
EP3504327B1 (en) 2016-08-24 2021-10-06 Sangamo Therapeutics, Inc. Engineered target specific nucleases
US20210047627A1 (en) * 2018-01-30 2021-02-18 Parkinson's Institute Crispr-based downregulation of alpha-synuclein expression as a novel parkinson's disease therapeutic

Also Published As

Publication number Publication date
WO2021067871A1 (en) 2021-04-08
TW202126681A (zh) 2021-07-16
AU2020356962A1 (en) 2022-04-14
KR20220071248A (ko) 2022-05-31
IL291827A (en) 2022-06-01
CA3156258A1 (en) 2021-04-08
WO2021067871A9 (en) 2021-05-06
US20220324928A1 (en) 2022-10-13
EP4041754A1 (en) 2022-08-17
JP2022551286A (ja) 2022-12-08

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